Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
Register:
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ClinicalTrials.gov |
Last refreshed on:
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30 March 2015 |
Main ID: |
NCT01601340 |
Date of registration:
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12/05/2012 |
Prospective Registration:
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Yes |
Primary sponsor: |
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Public title:
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Effects of HQK-1001 in Patients With Sickle Cell Disease
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Scientific title:
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A Randomized, Placebo-controlled, Phase 2 Study of HQK-1001 in Sickle Cell Disease |
Date of first enrolment:
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July 2012 |
Target sample size:
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77 |
Recruitment status: |
Terminated |
URL:
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http://clinicaltrials.gov/show/NCT01601340 |
Study type:
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Interventional |
Study design:
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Allocation: Randomized, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
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Phase:
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Phase 2
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Countries of recruitment
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Canada
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Egypt
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Jamaica
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Lebanon
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United States
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Contacts
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Name:
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Richard Ghalie, MD, MBA |
Address:
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Telephone:
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Email:
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Affiliation:
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HemaQuest Pharmaceuticals Inc. |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- Males and females between 12 and 60 years of age
- Diagnosis of SCD, type Hb SS or Hb S-B0 Thalassemia
- At least 1 episode of SCD pain crisis, acute chest syndrome, other acute SCD
complications, or leg ulcers in the 12 months prior to screening
- Not being treated with Hydroxyurea (HU); if HU treatment has been previously
administered and then discontinued, at least 3 months must have elapsed since last
dose of HU
- If subject has been transfused in the 3 months prior to screening, then Hb A level <
20% at screening
- Baseline Hb F level obtained within 14 days prior to randomization
- Able to swallow tablets
- Able and willing to give informed consent and/or assent
- If subject is a woman of child-bearing potential (WCBP), she must have a negative
serum pregnancy test within 14 days of first dose of HQK-1001 and a negative urine
pregnancy test prior to dosing on Day 1
- If a subject is a WCBP, she must agree to use an effective form of contraception
starting at screening and for one month after HQK-1001 discontinuation
- Sexually active male subjects who have not had a vasectomy must agree to use latex
condoms with WCBP partners or ensure that their partner(s) use an effective form of
contraception starting at screening and for one month after HQK-1001 discontinuation.
Exclusion Criteria:
- Assigned to a regular transfusion program
- Use of erythropoiesis stimulating agents within 90 days prior to screening
- An SCD pain crisis or SCD-related acute complication within 3 weeks prior to
randomization
- More than 5 SCD pain crisis or SCD-related acute complications within 12 months prior
to screening
- Pulmonary hypertension requiring therapy
- ALT or AST > 3x ULN
- Serum creatinine > 1.5x ULN
- Serum amylase levels > 1.5x ULN
- Serum lipase level > 1.5x ULN
- A serious, concurrent illness that would limit ability to complete or comply with the
study requirements
- An acute illness (e.g., febrile, GI, respiratory) within 72 hours prior to screening
- History of syncope, clinically significant dysrhythmias or resuscitation from sudden
death due to SCD-related complication
- Symptomatic peptic ulcer, hiatus hernia, or gastroesophageal reflux disease (GERD)
- History of pancreatitis
- Chronic opiate use, which, in the view of the investigator, could confound evaluation
of an investigational drug
- Current abuse of alcohol or drugs
- Use of another investigational agent within 4 weeks or 5 half-lives, whichever is
longer, prior to screening
- Currently pregnant or breast feeding a child
- Known infection with HIV-1
- Infection with hepatitis B or hepatitis C, such that subjects are currently on
anti-viral therapy or will be placed on therapy
Age minimum:
12 Years
Age maximum:
60 Years
Gender:
Both
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Health Condition(s) or Problem(s) studied
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Sickling Disorder Due to Hemoglobin S
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Sickle Cell Disease
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Hemoglobin S Disease
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Sickle Cell Anemia
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Sickle Cell Disorders
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Intervention(s)
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Drug: Placebo
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Drug: HQK-1001
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Primary Outcome(s)
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Change from baseline in % fetal hemoglobin
[Time Frame: Day 1 through Week 48]
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Secondary Outcome(s)
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Incidence and number of SCD pain crises and SCD-related complications
[Time Frame: Day 1 through Week 52]
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Safety measured by the frequency and severity of adverse events, and changes from baseline in vital signs, electrocardiogram (ECG) monitoring, and laboratory assessments
[Time Frame: Day 1 through Week 52]
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HQK-1001 pharmacokinetic parameters
[Time Frame: 1 hour prior to, and 2 hours following morning dose on Weeks 12, 24 and 48]
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Change in FACIT Fatigue Scale results
[Time Frame: Day 1 and Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, and 48]
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Subject reported daily pain scale scores and analgesic use
[Time Frame: 7 consecutive days following clinic visits at Day 1, and Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, and 48]
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Secondary ID(s)
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HQP 1001-SCD-007
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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